- Akashi Therapeutics on Monday suspended its Phase 1b/2a trial for HT-100, an experimental treatment for Duchenne muscular dystrophy (DMD), after a patient experienced "serious, life-threatening health issues."
- The suspension is yet another blow to hopes for a DMD treatment. Earlier in January, the FDA rejected Biomarin's drisapersen for inadequate efficacy evidence. Just a day after issuing its rejection, an FDA review strongly criticized clinical trial data for a competing treatment being developed by Sarepta.
- DMD is a genetic degenerative muscular disease mostly affecting male children and adolescents. Life expectancy for those suffering from DMD is usually less than 30 years, and there is no known cure.
Akashi suspended both the dosing and enrollment in its HT-100 trial. The patient experiencing serious health issues had received the highest dose in the study, 60μg/kg/d, for roughly two weeks. Other patients in previous cohorts had taken lower doses over a period of 11 to 19 months with no adverse events reported.
Akashi still is not certain whether the serious health problems in the patient resulted from HT-100 dosing or other factors.
Duchenne patients communities have been hoping to see a treatment approved for use in the U.S, as none currently are. One drug, made by PTC Therapeutics, has received a conditional approval in the E.U.
But those hopes have dimmed significantly after both Biomarin's drisapersen and Sarepta's eteplirsen ran into regulatory trouble from the FDA. Drisapersen could still be approved by the European Medicines Agency. As for Sarepta, the FDA advisory committee meeting for its drug was originally scheduled for January 22nd, but was delayed due to a major East Coast snow storm.
Akashi intends to restart its trial once the causes of the patient's health issues are more fully investigated. The timing of this investigation, and of discussions with the FDA, is not clear.